EP3355892A4 - Verfahren zur behandlung von muskeldystrophie - Google Patents

Verfahren zur behandlung von muskeldystrophie Download PDF

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Publication number
EP3355892A4
EP3355892A4 EP16852633.3A EP16852633A EP3355892A4 EP 3355892 A4 EP3355892 A4 EP 3355892A4 EP 16852633 A EP16852633 A EP 16852633A EP 3355892 A4 EP3355892 A4 EP 3355892A4
Authority
EP
European Patent Office
Prior art keywords
methods
treating muscle
muscle dystrophy
dystrophy
treating
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Withdrawn
Application number
EP16852633.3A
Other languages
English (en)
French (fr)
Other versions
EP3355892A1 (de
Inventor
Edward M. KAYE
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sarepta Therapeutics Inc
Original Assignee
Sarepta Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Priority to EP19207672.7A priority Critical patent/EP3653216A1/de
Publication of EP3355892A1 publication Critical patent/EP3355892A1/de
Publication of EP3355892A4 publication Critical patent/EP3355892A4/de
Withdrawn legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/58—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids containing heterocyclic rings, e.g. danazol, stanozolol, pancuronium or digitogenin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/06—Anabolic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Endocrinology (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
EP16852633.3A 2015-09-30 2016-09-29 Verfahren zur behandlung von muskeldystrophie Withdrawn EP3355892A4 (de)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP19207672.7A EP3653216A1 (de) 2015-09-30 2016-09-29 Verfahren zur behandlung von muskeldystrophie

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562235477P 2015-09-30 2015-09-30
US201662278866P 2016-01-14 2016-01-14
US201662293235P 2016-02-09 2016-02-09
US201662299952P 2016-02-25 2016-02-25
PCT/US2016/054534 WO2017059131A1 (en) 2015-09-30 2016-09-29 Methods for treating muscular dystrophy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
EP19207672.7A Division EP3653216A1 (de) 2015-09-30 2016-09-29 Verfahren zur behandlung von muskeldystrophie

Publications (2)

Publication Number Publication Date
EP3355892A1 EP3355892A1 (de) 2018-08-08
EP3355892A4 true EP3355892A4 (de) 2019-05-22

Family

ID=58424307

Family Applications (2)

Application Number Title Priority Date Filing Date
EP19207672.7A Withdrawn EP3653216A1 (de) 2015-09-30 2016-09-29 Verfahren zur behandlung von muskeldystrophie
EP16852633.3A Withdrawn EP3355892A4 (de) 2015-09-30 2016-09-29 Verfahren zur behandlung von muskeldystrophie

Family Applications Before (1)

Application Number Title Priority Date Filing Date
EP19207672.7A Withdrawn EP3653216A1 (de) 2015-09-30 2016-09-29 Verfahren zur behandlung von muskeldystrophie

Country Status (9)

Country Link
US (2) US20190054113A1 (de)
EP (2) EP3653216A1 (de)
JP (1) JP2018529715A (de)
CN (1) CN108738310A (de)
BR (1) BR112018006445A2 (de)
EA (1) EA201890811A1 (de)
HK (1) HK1258823A1 (de)
IL (1) IL258211A (de)
WO (1) WO2017059131A1 (de)

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PL1766010T3 (pl) 2004-06-28 2011-07-29 Univ Western Australia Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania
BR122023023194A2 (pt) 2009-11-12 2023-12-26 The University Of Western Australia Oligonucleotídeo antissentido, composição e respectivo uso
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
EA202090946A3 (ru) 2013-03-14 2021-07-30 Сарепта Терапьютикс, Инк. Композиции, обеспечивающие пропускание экзонов, для лечения мышечной дистрофии
NZ631289A (en) 2013-03-15 2017-08-25 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
US11083737B1 (en) * 2016-05-27 2021-08-10 Ptc Therapeutics, Inc. Methods of administering deflazacort therapy
CN110381980A (zh) 2017-01-06 2019-10-25 艾维迪提生物科学有限责任公司 核酸-多肽组合物以及诱导外显子跳读的方法
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
BR112020026542A2 (pt) * 2018-06-26 2021-04-06 Nippon Shinyaku Co., Ltd. Composição que compreende oligonucleotídeo antissenso e uso da mesma para tratamento de distrofia muscular de duchenne
SG11202108757XA (en) * 2019-03-28 2021-10-28 Sarepta Therapeutics Inc Methods for treating muscular dystrophy with casimersen
CA3153902A1 (en) 2019-10-16 2021-04-22 Pardis SABETI Engineered muscle targeting compositions
US20230159949A1 (en) 2020-07-22 2023-05-25 The Broad Institute, Inc. Engineered muscle targeting compositions
CN116507338A (zh) * 2020-09-21 2023-07-28 雷沃根生物医药有限公司 NF-κB介导的疾病的治疗
US20250170259A1 (en) 2022-03-03 2025-05-29 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
WO2023192904A1 (en) * 2022-03-30 2023-10-05 Biomarin Pharmaceutical Inc. Dystrophin exon skipping oligonucleotides

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CH445129A (fr) 1964-04-29 1967-10-15 Nestle Sa Procédé pour la préparation de composés d'inclusion à poids moléculaire élevé
US3459731A (en) 1966-12-16 1969-08-05 Corn Products Co Cyclodextrin polyethers and their production
US3426011A (en) 1967-02-13 1969-02-04 Corn Products Co Cyclodextrins with anionic properties
US3453257A (en) 1967-02-13 1969-07-01 Corn Products Co Cyclodextrin with cationic properties
US3453259A (en) 1967-03-22 1969-07-01 Corn Products Co Cyclodextrin polyol ethers and their oxidation products
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
WO1986005519A1 (en) 1985-03-15 1986-09-25 James Summerton Polynucleotide assay reagent and method
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US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
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KR0166088B1 (ko) 1990-01-23 1999-01-15 . 수용해도가 증가된 시클로덱스트린 유도체 및 이의 용도
EP1251170A3 (de) 1992-07-17 2002-10-30 Ribozyme Pharmaceuticals, Inc. Vergahren und Reagenz zur Behandlung von NF-kappaB abhängiger Tierischen Krankheiten
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US8299206B2 (en) 2007-11-15 2012-10-30 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
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CN105779453A (zh) * 2008-10-24 2016-07-20 萨雷普塔治疗公司 用于dmd的多外显子跳跃组合物
KR102182663B1 (ko) 2010-05-28 2020-11-25 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
NZ631289A (en) * 2013-03-15 2017-08-25 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
EA201990558A3 (ru) * 2013-04-20 2021-02-26 Рисёрч Инститъют Эт Нэйшнвайд Чилдрен'С Хоспитал Доставка нацеленных на экзон 2 полинуклеотидных конструкций u7snrna при помощи рекомбинантного аденоассоциированного вируса

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ANONYMOUS: "Sarepta Therapeutics Reports Long-Term Outcomes through 168 Weeks from Phase IIb Study of Eteplirsen en in Duchenne Muscular Dystrophy", 12 January 2015 (2015-01-12), XP055580695, Retrieved from the Internet <URL:http://investorrelations.sarepta.com/static-files/be998766-867b-4104-8531-35f5b6046496> [retrieved on 20190412] *
JERRY R. MENDELL ET AL: "Eteplirsen for the treatment of Duchenne muscular dystrophy", ANNALS OF NEUROLOGY, vol. 74, no. 5, 10 September 2013 (2013-09-10), pages 637 - 647, XP055135317, ISSN: 0364-5134, DOI: 10.1002/ana.23982 *
See also references of WO2017059131A1 *

Also Published As

Publication number Publication date
HK1258823A1 (zh) 2019-11-22
US20210008095A1 (en) 2021-01-14
EP3653216A1 (de) 2020-05-20
IL258211A (en) 2018-05-31
JP2018529715A (ja) 2018-10-11
EP3355892A1 (de) 2018-08-08
WO2017059131A1 (en) 2017-04-06
EA201890811A1 (ru) 2018-09-28
CN108738310A (zh) 2018-11-02
BR112018006445A2 (pt) 2018-12-11
US20190054113A1 (en) 2019-02-21

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